Efficacy Study of the Use of Sequential DFP-DFO Versus DFP

NCT ID: NCT00733811

Last Updated: 2008-08-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 213 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2000-09-30
• Study Completion Date: 2008-01-31

Brief Summary

Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published.

This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.

Detailed Description

The trial was designed as a multicentre randomised open-label trial with blinded data management and data analyses, to assess whether either treatment was superior to the other. The trial was performed on behalf of the Italian Society for the study of Thalassemia and Haemoglobinopathies (SoSTE). The investigators initiated, carried out, and controlled the trial, which was conducted without influence of the non-commercial sponsor.16

Conditions

Beta-Thalassemia; Thalassemia Major

Keywords

thalassemia major; chelation treatment; secondary hemochromatosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

1

Sequential treatment including DFP at 75 mg/kg, divided into three oral daily doses, for four days per week and DFO by subcutaneous infusions (8-12h) at 50 mg/kg/day for the remaining three days per week

Group Type: EXPERIMENTAL

Deferiprone (DFP) and Deferoxamine (DFO)

Intervention Type: DRUG

Sequential treatment including DFP at 75 mg/kg for four days per week and DFO by subcutaneous infusions (8-12h) at 50mg/kg/day for the remaining three days per week

2

Deferiprone alone at 75 mg/kg divided into three oral daily doses

Group Type: ACTIVE_COMPARATOR

Deferiprone (DFP)

Intervention Type: DRUG

DFP alone at 75 mg/kg divided into three oral daily doses

Interventions

Deferiprone (DFP) and Deferoxamine (DFO)

Sequential treatment including DFP at 75 mg/kg for four days per week and DFO by subcutaneous infusions (8-12h) at 50mg/kg/day for the remaining three days per week

Intervention Type: DRUG

Deferiprone (DFP)

DFP alone at 75 mg/kg divided into three oral daily doses

Intervention Type: DRUG

Other Intervention Names

DFP (Apotex, Canada); DFO (Biofutura Pharma S.p.A.,Italy); DFP (Apotex, Canada)

Eligibility Criteria

Inclusion Criteria

• Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age

Exclusion Criteria

• Known intolerance to one of the trial treatments
• Platelet count < 100,000/mm3 or or leukocyte count < 3,000/mm3
• Severe liver damage indicated by ascites
• Heart failure

• Minimum Eligible Age: 13 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Azienda Ospedaliera V. Cervello

OTHER

Sponsor Role: lead

Responsible Party

AO V Cervello

Principal Investigators

AURELIO MAGGIO, M.D.

Role: STUDY_CHAIR

Azienda Ospedaliera V. Cervello

Locations

Ao V. Cervello

Palermo, , Italy

Countries

Italy

References

Maggio A, Vitrano A, Capra M, Cuccia L, Gagliardotto F, Filosa A, Romeo MA, Magnano C, Caruso V, Argento C, Gerardi C, Campisi S, Violi P, Malizia R, Cianciulli P, Rizzo M, D'Ascola DG, Quota A, Prossomariti L, Fidone C, Rigano P, Pepe A, D'Amico G, Morabito A, Gluud C. Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial. Br J Haematol. 2009 Apr;145(2):245-54. doi: 10.1111/j.1365-2141.2009.07609.x. Epub 2009 Feb 19.

Reference Type: DERIVED

PMID: 19236376 (View on PubMed)

Other Identifiers

AOVCervello

Identifier Type: -

Identifier Source: org_study_id